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Updated: Jun 28, 2026

Multi-exon Skipping Using Cocktail Antisense Oligonucleotides in the Canine X-linked Muscular Dystrophy
Published on: May 24, 2016
Asymptomatic Becker muscular dystrophy in a family with a multiexon deletion
Verónica Ferreiro1, Florencia Giliberto, García M Noelia Muñiz
1Genetics Division, Clinical Hospital "José de San Martín," University of Buenos Aires, 2250 Paraguay, 1120 Buenos Aires, Argentina. vferreiro@ffyb.uba.ar
Abstract:
We report a Becker muscular dystrophy (BMD) family with one 5-year-old affected patient and a 69-year-old asymptomatic grandfather. Dystrophin gene multiplex polymerase chain reaction and multiplex ligation-dependant probe amplification analysis showed that both males carried an in-frame deletion of exons 45-55. Segregation analysis revealed two additional asymptomatic boys in this family. Our finding supports previous predictions that exons 45-55 are the optimal multiexon skipping target in antisense gene therapy to transform the severe Duchenne muscular dystrophy into the milder BMD, or even asymptomatic, phenotype.
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